Cogent Biosciences Announces Breakthrough Therapy Designation for Bezuclastinib in Combination with Sunitinib for Patients with Gastrointestinal Stromal Tumors (GIST)

Core Insights - Cogent Biosciences has received Breakthrough Therapy Designation from the FDA for bezuclastinib in combination with sunitinib for patients with imatinib-resistant Gastrointestinal Stromal Tumors (GIST) [2][3] - The company plans to submit the New Drug Application (NDA) under the Real-Time Oncology Review (RTOR) program, aiming for completion by April 2026 [1][3] Clinical Trial Results - The PEAK trial demonstrated a 50% reduction in the risk of disease progression or death compared to the current standard of care, with a hazard ratio of 0.50 (95% CI: 0.39 – 0.65) [2] - Median progression-free survival (mPFS) was reported as 16.5 months for the bezuclastinib combination versus 9.2 months for sunitinib monotherapy [2] Future Plans - Cogent intends to present full results from the PEAK trial at a major medical meeting in the first half of 2026 [4] - The company expects to initiate a Phase 2 trial in mid-2026 to investigate the bezuclastinib and sunitinib combination for first-line GIST patients with exon 9 mutations [4] Company Overview - Cogent Biosciences focuses on developing precision therapies for genetically defined diseases, with bezuclastinib being a selective tyrosine kinase inhibitor targeting the KIT D816V mutation [5] - The company is also developing a portfolio of targeted therapies aimed at various mutations, including FGFR2/3, ErbB2, PI3Kα, KRAS, and JAK2 [5]